Table 1.

Published reports of brain tumor patients treated with BRAF-targeted therapy

Brain tumor type	V600E incidence	Agent	Combination therapy	No. of patients	Effect	Additional information	Reference
Pilocytic astrocytoma	9% [22]	Dabrafenib		1	Resolution of metastatic disease, decrease in primary tumor		[23]
		Dabrafenib	Trametinib	1	Substantial rPR, cCR		[24]
		Vemurafenib		2	1 PR		[25]
Pediatric low-grade astrocytoma	20–43% [26]	Dabrafenib		32	2 CR; 11 PR; 13 SD ≥ 6 months	V600 mutation	[27, 28]
		Selumetinib		7	2 PR		[29]
		Selumetinib		3	2 PR	1 Patient with KIAA1549-BRAF fusion: rapid progression	[30]
Pediatric high-grade astrocytoma	12–27% [31]	Dabrafenib	Trametinib	3	3 PR (20, > 23, > 32 months)		[32]
		Vemurafenib		1	Transient PR		[33]
Adult high-grade astrocytoma	3% [34]	Dabrafenib	NovoTTF-100A	1	CR for > 2 years	Tumor resulting from GG	[35]
		Dabrafenib	Trametinib	31	1 CR; 7 PR Median PFS 1.9 months. Median OS 11.7 months	5 of responding patients: DOR of ≥ 12 months	[36]
		Dabrafenib	Trametinib	2	PR for 3 and 11 months	1 patient treated in the first-line setting	[37]
		Dabrafenib	Trametinib	1	No therapeutic benefit	Concurrent gain of function mutation of EGFR	[38]
		Dabrafenib	Trametinib	1	SD for > 16 months		[39]
		Vemurafenib		11	1 PR; 5 SD (2 for > 1 year)		[25]
Pleomorphic xanthoastrocytoma	50% [40]-66% [22]-78% [26]	Dabrafenib	Trametinib	1	Substantial rPR, cCR	Grade II PXA	[24]
		Dabrafenib	Trametinib	1	Transient radiographic and clinical response	Grade III PXA	[38]
		Dabrafenib	Trametinib	1	PR for 14 months, than clinical and radiographic progression	Grade III PXA	[39]
		Dabrafenib	Trametinib + chloroquine	1	SD for > 2.5 years	Grade III PXA	[41]
		Vemurafenib		7	1 CR; 2 PR; 3 SD	Grade II PXAs	[25]
		Vemurafenib		4	1 PR; 2 SD Median PFS 5 months Median OS 8 months	Grade II PXAs	[42]
Ganglioglioma	9–18% (adult) [22] -49% (pediatric) [26]	Dabrafenib	Trametinib	1	CR for > 6 months	Anaplastic GG	[43]
		Dabrafenib	Trametinib	1	PR; SD for > 6 months	Anaplastic GG	[44]
		Dabrafenib	Trametinib	1	Substantial PR		[24]
		Vemurafenib		2	1 PR; SD for > 20 months	Pediatric patients	[33]
		Vemurafenib		1	PR; SD for > 6 months	Cervicomedullary GG	[45]
		Vemurafenib		3	1 PR		[25]
		Vemurafenib		1	PR; SD for > 33 months	Tumor of spinal cord Patient stopped treatment after a year	[46]
		Vemurafenib		1	PR; SD for 1 year	Pediatric tumor	[47]
		Vemurafenib	Cobimetinib	1	CR for > 16 months	Tumor with acquired resistance to vemurafenib	[48]
		Vemurafenib	Vinblastine	1	CR for > 12 weeks	Pediatric, brainstem GG	[49]
Papillary craniopharyngioma	95% [50]	Dabrafenib		1	PR; SD for > 21 months		[51]
		Dabrafenib	Trametinib	1	Substantial rPR, cCR		[24]
		Dabrafenib	Trametinib	1	PR for > 7 months		[52]
Spindle cell oncocytoma	n/a	Dabrafenib/ vemurafenib	Trametinib/ cobimetinib	1	PR; SD for > 24 months	Patient developed panniculitis	[53]

cCR clinical complete response, CR complete response, DOR duration of response, GG ganglioglioma, OS overall survival, PFS progression-free survival, PR partial response, PXA pleomorphic xanthoastrocytoma, rPR radiological partial response, SD stable disease